Summit Therapeutics announced today that it will proceed with the planned extension phase of PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval.
This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data Monitoring Committee and its support of Summit’s plans to extend the clinical trial.
The proposed extension phase will allow Summit to gather important long term safety and efficacy data of ezutromid in people with Duchenne that will likely be included in the data package for future applications as Summit continues to pursue regulatory approval of ezutromid.
Read the announcement from Summit:
· Decision follows interim safety review by PhaseOut DMD’s independent Data Monitoring Committee
Oxford, UK, 27 March 2017 – Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, announces that it will proceed with the planned extension phase of PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval. This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data Monitoring Committee (‘DMC’) and its support of Summit’s plans to extend the clinical trial.
Summit has now applied for regulatory approval to extend PhaseOut DMD from the UK Medicines and Healthcare products Regulatory Agency and Ethics Committee, and has submitted the necessary regulatory updates to the US Food and Drug Administration. These submissions are intended to facilitate the transition of patients participating in PhaseOut DMD onto an open-label extension phase at the end of the initial 48-weeks of dosing with ezutromid without a cessation in dosing. The extension phase will be used to gather long term safety and efficacy data and is expected to last until ezutromid either receives marketing approval in the relevant country or its development is discontinued.
“The proposed extension phase will allow us to gather important long term safety and efficacy data of ezutromid in patients with DMD that we believe will comprise part of a data package necessary for future applications for regulatory approval of ezutromid,” Ralf Rosskamp, MD, Chief Medical Officer of Summit commented. “While the PhaseOut DMD trial continues with the aim of establishing proof of concept for ezutromid, we are pleased to see that the DMC supports the trial’s extension based on their review of the safety and tolerability data to date, which includes data from patients dosed over longer periods of time than have previously been tested.”
In addition to the extension phase of the trial, the regulatory submissions also include the addition of a safety arm to allow for the enrolment of patients who have previously taken part in Phase 1 clinical trials of ezutromid, but who did not meet the inclusion criteria for PhaseOut DMD. Although regulatory approval for the safety arm is being sought, the timing for inclusion of these patients into the trial is to be determined.
About PhaseOut DMD
PhaseOut DMD aims to provide proof of concept for ezutromid and utrophin modulation by measuring muscle fat infiltration, as well as by measuring utrophin protein and muscle fibre regeneration in muscle biopsies. The primary endpoint of the open-label trial is the change from baseline in magnetic resonance imaging parameters related to fat infiltration and inflammation of the leg muscles. Exploratory endpoints include the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes. PhaseOut DMD is a 48-week open-label trial expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays at sites in the UK and the US. Each patient will receive two biopsies, one at baseline and the second either at 24 or 48 weeks. Further information is available at: https://clinicaltrials.gov/ct2/show/NCT02858362 and www.utrophintrials.com.
About Utrophin Modulation in DMD
DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company’s lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration (‘FDA’) and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA.